Olivas-Mazón Raquel, Bueno David, Sisinni Luisa, Mozo Yasmina, Casado-Abad Gema, Martínez Antonio Pérez
Pediatric Hemato-Oncology Department, La Paz University Hospital, Madrid, Spain.
Pharmacy Department, La Paz University Hospital, Madrid, Spain.
Eur J Haematol. 2022 Nov;109(5):474-482. doi: 10.1111/ejh.13828. Epub 2022 Jul 21.
To compare the outcomes of treosulfan-based vs busulfan-based conditioning regimens in allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric patients.
Retrospective study of all consecutive patients (2012-2019) treated with allogenic HSCT and treosulfan- or busulfan-based conditioning regimens at a single center.
A total of 101 HSCT were included: 66 HSCT with busulfan and 35 with treosulfan. In malignant diseases (n = 62), busulfan-based conditioning was more commonly employed than treosulfan: 82.3% vs 17.7%. However, the use of treosulfan for malignant diseases increased over time: 6.5% of HSCT in 2012-2015 vs 29% of HSCT in 2015-2019 (p = .02). The cohort of treosulfan had more children under 1-year of age than the busulfan cohort (31 vs 13%; p = .033). The percentage of patients who received serotherapy was 73 and 89% in the nonmalignant and malignant groups, respectively. The engraftment, time to neutrophil, and platelet engraftment were not significantly different between the busulfan and the treosulfan cohorts. Rate of grade II-IV acute GvHD was significantly higher in the busulfan cohort than the treosulfan cohort (39% vs 15%; p = .016). No differences were observed in endothelial damage complications, chronic GvHD, relapse, overall survival, and transplant-related mortality.
Busulfan-based conditioning regimens are used more frequently for children undergoing allogenic HSCT, but treosulfan-based conditioning is gaining acceptance. Treosulfan-based conditioning is associated with lower rates of acute GvHD, and no significant differences on overall survival were observed compared with busulfan.
比较小儿患者异基因造血干细胞移植（HSCT）中基于曲奥舒凡与基于白消安的预处理方案的疗效。
对某单一中心2012年至2019年接受异基因HSCT及基于曲奥舒凡或白消安预处理方案的所有连续患者进行回顾性研究。
共纳入101例HSCT：66例采用白消安预处理，35例采用曲奥舒凡预处理。在恶性疾病患者（n = 62）中，基于白消安的预处理方案比基于曲奥舒凡的方案使用更为普遍：分别为82.3%和17.7%。然而，随着时间推移，曲奥舒凡在恶性疾病中的使用有所增加：2012年至2015年HSCT的6.5%，2015年至2019年为29%（p = 0.02）。曲奥舒凡组1岁以下儿童比白消安组更多（31%对13%；p = 0.033）。非恶性和恶性组接受血清疗法的患者百分比分别为73%和89%。白消安组和曲奥舒凡组之间的植入、中性粒细胞恢复时间和血小板植入情况无显著差异。白消安组II-IV级急性移植物抗宿主病（GvHD）发生率显著高于曲奥舒凡组（39%对15%；p = 0.016）。在内皮损伤并发症、慢性GvHD、复发、总生存率和移植相关死亡率方面未观察到差异。
接受异基因HSCT的儿童更频繁使用基于白消安的预处理方案，但基于曲奥舒凡的预处理方案正逐渐被接受。基于曲奥舒凡的预处理方案与较低的急性GvHD发生率相关，与白消安相比，总生存率无显著差异。
